European Commission Approves Spark Therapeutics’ LUXTURNA

Spark Therapeutics (NASDAQ:ONCE), a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced that the European Commission has granted marketing authorization for LUXTURNA (voretigene neparvovec), a one-time gene therapy for the treatment of adult and pediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.

As quoted in the press release:

RPE65-mediated inherited retinal disease (IRD) is a progressive condition leading to total blindness in most patients. This authorization is valid in all 28-member states of the EU, as well as Iceland, Liechtenstein and Norway. LUXTURNA is the first gene therapy for a genetic disease that has received regulatory approval in both the U.S. and EU.

“The historic approval of LUXTURNA in Europe furthers our mission to challenge the inevitability of genetic disease around the world. Following the launch of LUXTURNA in the United States earlier this year, this decision makes LUXTURNA the first gene therapy for a genetic disease approved in both the U.S. and EU, a promising milestone for the many people living with genetic disease around the world,” said Ron Philip, chief commercial officer at Spark Therapeutics. “Having worked closely with the global IRD community on the development of LUXTURNA, we are proud to be able to share the first gene therapy treatment option for an inherited retinal disease (IRD) with appropriate patients in Europe, in collaboration with Novartis.”

Click here to read the full press release.